GAD65 + VGAT gene therapy

Neurgain Technologies is developing an investigational AAV-based gene therapy (utilizing AAV9 or Anc80 vectors) designed to treat neuropathic pain and chronic spasticity. The therapy delivers two transgenes, glutamic acid decarboxylase 65 (GAD65) and vesicular GABA transporter (VGAT), specifically to dorsal horn neurons in the spinal cord. By increasing GABA synthesis via GAD65 and enhancing its synaptic vesicular packaging and release via VGAT, the treatment aims to restore GABAergic inhibition in pain-signaling nociceptive neurons. A key feature of this approach is the proprietary spinal subpial delivery technique, which provides precise, targeted expression across multiple spinal cord segments. Preclinical studies have demonstrated long-lasting pain reversal without motor weakness or loss of normal sensation.

Other names

GAD65 + VGAT combination gene therapyGene therapy (GAD65 + VGAT)